Savara Inc. advances MOLBREEVI BLA with FDA Priority Review for autoimmune PAP treatment. PDUFA date: Aug 22, 2026. Backed by Phase 3 IMPALA-2 trial, it shows improved lung function & quality of life in rare lung disease.
Edited and Written By: Pharmacally Medical News Desk
Assisted By: Samiksha Jadhav, BPharm
Reviewed By: Pharmacally Editorial Team
Savara Inc., a clinical-stage biopharmaceutical company specializing in rare respiratory diseases, announced a major advancement in treating autoimmune pulmonary alveolar proteinosis (PAP). The U.S. Food and Drug Administration (FDA) has accepted and filed for review the Biologics License Application (BLA) for MOLBREEVI, positioning it as a potential therapy for patients with this debilitating condition. With Priority Review granted, the FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of August 22, 2026.
Matt Pauls, Chair and Chief Executive Officer of Savara, highlighted the significance of this milestone: “The FDA’s filing of the BLA marks another significant milestone for Savara and the autoimmune PAP community and brings us one step closer to a potential approval in the U.S. in August of this year.” He emphasized the robust data supporting MOLBREEVI, noting it demonstrates improvements in pulmonary gas transfer, quality of life, and clinical symptoms for this rare lung disease. Pauls also expressed gratitude for the FDA’s constructive feedback throughout development.
The BLA for MOLBREEVI relies primarily on data from Savara’s pivotal Phase 3 IMPALA-2 trial (NCT04544293), a 48-week, randomized, double-blind, placebo-controlled study in approximately 160 autoimmune PAP patients across 18 countries. IMPALA-2 met its primary endpoint with statistically significant improvement in percent predicted diffusing capacity for carbon monoxide (DLCO) at Week 24 versus placebo, alongside sustained benefits at Week 48, enhancements in St. George’s Respiratory Questionnaire scores, and improved exercise capacity (peak METs).
MOLBREEVI has already received multiple regulatory recognitions, underscoring its potential:
- Fast Track and Breakthrough Therapy Designations from the FDA.
- Orphan Drug Designation from both the FDA and European Medicines Agency (EMA).
- Innovation Passport (IP) and Promising Innovative Medicine (PIM) designations from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA).
These incentives highlight MOLBREEVI’s promise for a disease with limited treatment options.
Autoimmune Pulmonary Alveolar Proteinosis (PAP)
Autoimmune PAP is a rare lung disorder affecting the alveoli, the tiny air sacs where gas exchange occurs. Normally, surfactant a mix of proteins and lipids coats the alveoli to keep them open. Alveolar macrophages, immune cells in the lungs, clear excess surfactant using stimulation from granulocyte-macrophage colony-stimulating factor (GM-CSF).
In autoimmune PAP, the body produces autoantibodies that neutralize GM-CSF. This impairs macrophages, leading to surfactant buildup that blocks gas transfer. Patients experience progressive shortness of breath, cough, and fatigue. Acute episodes may include fever, chest pain, or hemoptysis (coughing up blood), often from secondary infections. Over time, complications like lung fibrosis can arise, sometimes necessitating a lung transplant.
Current management relies on whole lung lavage an invasive procedure or supportive care, with no approved pharmacologic therapies. MOLBREEVI aims to address the root cause by targeting the GM-CSF pathway.
Reference
Savara Announces the U.S. Food and Drug Administration (FDA) Filed the MOLBREEVI* Biologics License Application (BLA) in Autoimmune Pulmonary Alveolar Proteinosis (Autoimmune PAP), 20 February 2026, https://investors.savarapharma.com/news/news-details/2026/Savara-Announces-the-U-S–Food-and-Drug-Administration-FDA-Filed-the-MOLBREEVI-Biologics-License-Application-BLA-in-Autoimmune-Pulmonary-Alveolar-Proteinosis-Autoimmune-PAP/default.aspx
Clinical Trial of Inhaled Molgramostim Nebulizer Solution in Autoimmune Pulmonary Alveolar Proteinosis (aPAP) (IMPALA-2), ClinicalTrials.gov ID NCT04544293, https://clinicaltrials.gov/study/NCT04544293
Bruce C. Trapnell et al, Phase 3 Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis, N Engl J Med 2025;393:764-773, DOI: 10.1056/NEJMoa2410542, https://www.nejm.org/doi/full/10.1056/NEJMoa2410542